Agios Pharmaceuticals announced positive results from the ACTIVATE-Kids Phase 3 study, the first to demonstrate the efficacy of mitapivat, an oral therapy, for children aged 1 to under 18 with ...

– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...

BOSTON, Mass. (Ivanhoe Newswire) – PK deficiency is a genetic blood disorder that causes red blood cells to die prematurely. Regular blood transfusions have been the primary treatment for people with ...

Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...

Q4 2025 Management View CEO Brian Goff emphasized the company's 2026 strategic priorities, stating, "we're focused on executing a high-impact launch of AQVESME for the treatment of thalassemia in the ...

CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined ...

Credit: Shutterstock. Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes. The Food and Drug Administration (FDA) has accepted for Priority ...

The FDA has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The Food and Drug Administration (FDA) has approved Pyrukynd ® (mitapivat) ...

On Thursday, Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

Agios Pharmaceuticals Inc. has started a phase I dosing study of its lead candidate for inborn errors of metabolism, AG-348. The experimental treatment for pyruvate kinase (PK) deficiency, a rare ...

Cambridge, Massachusetts-based Agios will market its new product, a twice-daily tablet, under the name “Pyrukynd.” The FDA’s Thursday approval covers use of the drug in adults who have an inherited ...